Advanced therapies promise to change the world of medicine, provided they can reach the market. Thanks to techniques such as gene and cell therapy, tissue engineering, stem cells and Car-Ts, new frontiers are opening up in medical research and in improving patients’ quality of life, but entering this field is far from easy. The path to bring a valuable scientific idea to market is, in fact, particularly demanding and involves many difficult challenges: taking the necessary technical and scientific expertise for granted, there is a labyrinth of regulations, a highly competitive market, very high development costs and, of course, the need for adequate funding to reach the market. Maria Luisa Nolli has always been involved in technology transfer: even before embarking on her entrepreneurial career (she is currently CEO of NCNbio, a company she founded more than 10 years ago that works on industrial technology transfer in biotechnology), she was involved in this activity within the Dow Chemical group: ‘At that time, I worked within the research centre, whose objective was to identify leads to be brought to market. Even then, from department to department, there was a transfer of technology along the drug development chain. Subsequently, this process became more central and was applied to a wider variety of fields, including biotechnology’. We asked the entrepreneur to tell us how this process applies to the field of advanced therapies (Atmp, Advanced therapy medicinal product) and – most importantly – what it takes to govern it.
What is the role of technology transfer along the drug supply chain, in particular of Atmps?
In this chain, regardless of the nature of the drugs, whether synthetic or biotechnological (antibodies, proteins or advanced therapies such as cellular drugs) certain steps are shared: we are talking about research and development, pre-clinical, manufacturing, clinical phases, market access and then post-marketing with surveillance. In the specific field of biotechnological drugs, there are different types of technology transfer. Technology transfer is often regarded as a mechanical process, whereas I consider it to be something more articulated, running from the idea to the first proof of concept, to the first prototype and then to the final product. With regard to discoveries that need to be protected in terms of intellectual property, technology transfer is also functional to their valorisation, an objective that can be achieved in different ways. Today, the business model most commonly used by large companies involves forming alliances with universities and small biotech companies aimed at promoting the development of new products from scientific discoveries.
How does this process take place?
It all depends on the formula to be adopted. Technology transfer can consist of a university granting licences to a company for the use of scientific discoveries or product prototypes. Alternatively, a start-up or spinoff can be created around the scientific discovery through both public and private funding models. This creates interesting opportunities but requires the ability to put in place a set of instruments for this purpose . Right from the start, for instance, a start-up must have people representing all the sectors involved: scientific, organisational, administrative and sponsorship. It is not uncommon to come across university departments even with solid experience in the field of intellectual property but without any idea of how to manage the creation of start-ups from a governance point of view.
What are the most common criticalities?
Let me tell you about two episodes of failure that I happened to observe at close quarters and which I consider emblematic In the first case, the problem was the resistance of the scientists to share the project, the data and the results within their own start-up. It may sound absurd, but it happens. In the second, the critical aspect was the failure to replace management at the right time. Mind you, the figure of the scientist must always be present in an activity of this type, especially in the context of initiatives aimed at the development of sophisticated products such as advanced therapies, in which science must accompany all the steps, including post-marketing which, I would remind you, involves a surveillance of several years, 15 for advanced therapies. Moreover, these products are subject to continuous evolution, thus requiring constant scientific guidance. But one must always be aware of the general framework in which one moves and understand when it is time to make changes, albeit not radical but appropriate, depending on the stage of the process . Lacking this kind of sensitivity is one of the most costly mistakes one can make.
What figures are needed?
Skills in this area, especially in its most innovative expressions, must include a suitably modulated cocktail of science and business. Where are these skills to be found? In those who have been and are within the processes, have experienced them and continue to experience them. If with my NCNBio I still offered the solutions developed in Areta International, the company I founded in 2000 and sold in 2012, I would be obsolete today. It is crucial to share up-to-date knowledge in all teams. Unfortunately, today, non-specific consultancy often leads to taking shortcuts that end in failure. Alongside the products, business and management models are also changing. Let’s think of the first two Car-T therapies to reach the market, Kymriah and Yescarta, which are the result of the new approach mentioned above: the former was developed by Novartis partly thanks to collaboration with the University of Pennsylvania, while the latter is the result of Gilead’s acquisition of the biotech company Kite. Two billion-dollar deals. Neither Novartis nor Gilead chose to develop these products internally, but they formed strategic alliances to achieve this.
Also watch the AFI interview to Maria Luisa Nolli
